POS0221 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH TRAPS (TUMOR NECROSIS FACTOR RECEPTOR-ASSOCIATED PERIODIC SYNDROME) – INTERIM ANALYSIS OF THE RELIANCE REGISTRY
Authors: N. Blank, C. Schuetz, J. Henes, T. Kallinich, P. Oommen, M. Borte, M. Hufnagel, A. Janda, J. Weber-Arden, J. B. Kuemmerle-Deschner
Journal: Annals of the Rheumatic Diseases (2022)

Background Tumor necrosis factor receptor-associated periodic syndrome (TRAPS) is a rare hereditary autoinflammatory disease characterized by periodic fever as well as severe systemic and organ inflammation. Successful treatment was achieved with the interleukin-1β inhibitor canakinumab (CAN) in a pivotal phase 3 study, in which 45% of patients reached clinical remission after 16 weeks (primary endpoint) 1 . CAN has been approved and applied for the treatment of TRAPS patients since 2017 2 . Objectives The present study explores the long-term efficacy and safety of CAN under routine clinical practice conditions in pediatric (age ≥2 years) and adult TRAPS patients. Methods RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany. Patients with clinically confirmed diagnosis of TRAPS who routinely receive CAN are enrolled in order to evaluate efficacy and safety of CAN under standard clinical practice conditions at baseline and at 6-monthly intervals. Results The interim analysis of TRAPS patients enrolled by December 2021 includes baseline (N=19, 1 patient with atypical TRAPS) and preliminary 24-month data. Of these patients, N=12 (63%) were females and median age at baseline was 16 years (3–43 years). Preliminary results indicate stable remission by physicians´ assessment and laboratory parameters. Disease control by patients´ assessment showed no major changes over time (Table 1). In total, N=7 adverse drug reactions where observed, of which none was classified as severe. Table 1. Baseline characteristics and interim analysis data of patients with TRAPS. Baseline(N=19) 12 months(N=15) 24 months(N=8) Number (%) of patients in disease remission (physician assessment) 9 (53) 11 (79) 4 (67) Physician assessment of disease activity, percentage of absent/mild-moderate/severe rating 35 / 59 / 0 57 / 36 / 7 67 / 33 / 0 Patient´s assessment of current disease activity; 0–10, median (min; max) 1.5 (0; 8) 1.0 (0; 6) 0 (0; 7) Patient´s assessment of current fatigue; 0–10, median (min; max) 1.0 (0; 8) 1.0 (0; 8) 0 (0; 8) Number (%) of patients without impairment of social life by the disease 5 (56) 4 (44) 2 (100) Number (%) of patients with days absent from work/school during last 6 months 8 (44) 6 (40) 1 (14) CRP, median (mg/dl) 0.2 0.1 0.2 SAA, median (mg/dl) 0.5 0.4 0.4 ESR, median (mm/h) 7.0 5.5 6.0 SAE Number of events Incidence rate # per 100 patient years All types of SAE 9 24.2 SADR 0 0.0 CRP, c-reactive protein; SAA, serum amyloid A; ESR, erythrocyte sedimentation rate; #Numbers/percentage do not sum up to N=19/100%, due to unknown weight of some patients Conclusion Baseline characteristics and interim data of TRAPS patients are available from the RELIANCE study. Further interim and end-of-study data will be analyzed to assess efficacy and safety of long-term treatment as well as dosing effects in TRAPS patients. References [1]De Benedetti F, et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. N Engl J Med 2018;378:1908–19 [2]Ilaris, INN-canakinumab (europa.eu) Disclosure of Interests Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi, Catharina Schuetz: None declared, Jörg Henes Consultant of: Novartis, AbbVie, Sobi, Roche, Janssen, Boehringer-Ingelheim, Grant/research support from: Novartis, Roche, Tilmann Kallinich Consultant of: Sobi, Novartis, Roche, Grant/research support from: Novartis, Prasad Oommen Grant/research support from: Novartis, Michael Borte Grant/research support from: Pfizer, Shire, Markus Hufnagel Grant/research support from: Novartis, Ales Janda: None declared, Julia Weber-Arden Employee of: Novartis, J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi